Trials / Completed
CompletedNCT04470713
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 226 (actual)
- Sponsor
- Idorsia Pharmaceuticals Ltd. · Industry
- Sex
- All
- Age
- 0 Months
- Healthy volunteers
- Not accepted
Summary
This study is being conducted to better understand the natural course of GM1 gangliosidosis, GM2 gangliosidoses and Gaucher disease Type 2 (GD2). Information is planned to be gathered on at least 180 patients with GM1 gangliosidosis, GM2 gangliosidoses, and Gaucher Disease type 2. Retrospective data collection is planned for at least 150 deceased patients (Group A). Group B is for patients alive at the time of enrollment. In Group B it is planned to prospectively collect more comprehensive data from at least 30 patients. The purpose of this study is to collect relevant information for a adequate design of a potential subsequent research program in these diseases. In this study no therapy is being offered.
Conditions
- GM1 Gangliosidosis
- GM2 Gangliosidosis
- Gaucher Disease, Type 2
- Tay-Sachs Disease
- AB Variant Gangliosidosis GM2
- Sandhoff Disease
Timeline
- Start date
- 2019-07-31
- Primary completion
- 2021-10-30
- Completion
- 2021-10-30
- First posted
- 2020-07-14
- Last updated
- 2021-11-08
Locations
17 sites across 10 countries: United States, Belgium, Brazil, France, Germany, Italy, Portugal, Spain, Switzerland, United Kingdom
Source: ClinicalTrials.gov record NCT04470713. Inclusion in this directory is not an endorsement.